Rare Diseases and Conditions

These therapies replace missing or deficient proteins that allow individuals to lead healthy and more productive lives. The patients that rely on these therapies generally require regular infusions or injections throughout their lives. The diseases and conditions treated by plasma protein therapies are considered rare diseases because they affect a relatively small percent of the population. Most are genetic, chronic conditions.

Alpha-1 Proteinase Inhibitor therapies are used to treat:

Alpha-1 Antitrypsin Deficiency

Alpha-1 Antitrypsin Deficiency is one of the most common serious hereditary disorders in the world and can result in life-threatening liver disease in children and adults and lung disease in adults. It is often referred to as genetic emphysema.

C1 Esterase Inhibitor therapies are used to treat:

Hereditary Angioedema

Hereditary Angioedema is caused by a missing C1 esterase inhibitor protein (C1-INH), which helps regulate inflammation. Patients can experience edema (severe swelling) which can be fatal if the airway becomes obstructed.

Coagulation Factors are used to treat:

Hemophilia A

Hemophilia A is a heredity bleeding disorder that is caused by a lack of clotting factor VIII. As a result, individuals with this condition suffer from bleeding into joints and other complications. For the most part, men have hemophilia A as the defective gene is found on the X chromosome. A woman who has the defective gene is considered a carrier and any male offspring have a 50% chance of having hemophilia A and female offspring a 50% chance of being a carrier. It affects one in 10,000 people. With treatment, individuals are able to lead relatively normal lives.

Hemophilia B

Hemophilia B is a blood clotting disorder caused by a mutation of the Factor IX gene. It is rarer than hemophilia A and affected royal families in both Europe and Russia. It affects one in 25,000 men. Replacement of Factor IX through recombinant therapy allows individuals to lead relatively normal lives.

Von Willebrand Disease

The most common bleeding disorder, Von Willebrand affects about 1.25 million men and women worldwide, although it is estimated that as many as 3 million are undiagnosed. Common symptons include excessive menstrual bleeding and nosebleeds. There are several kinds of VWD and symptom severity varies. With proper treatment, individuals may live relatively normal lives.

Antithrombin III Deficiency

Antithrombin III is a protein that prevents blood clots from forming. It is caused by an abnormal gene that may lead to blood clots. Data on incidence is uncertain.

Immune Globulins are used to treat:

Primary Immunodeficiency Disease (PID)

PID is a genetic condition that prevents an individual's immune system from functioning properly. The World Health Organization (WHO) estimates that there may be as many as 150 PIDs. PID results a high susceptibility to infection and an inability to fight them with traditional antiobiotics.

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

CIDP is a rare autoimmune disorder that affects the peripheral nervous system of both children and adults. Nerves in the arms and legs may become weakened and lead to paralysis.

PPTA News

The article, “How Blood-Plasma Companies Target the Poorest Americans,” by H. Luke Shaefer and Analidis Ochoa, published in The Atlantic on March 15, was unfair to plasma donors as well as to individuals living with rare, genetic, and chronic diseases who rely on access to plasma protein therapies. Plasma donors are due our gratitude and respect, not sweeping negative characterizations.

Highlights from the 2018 IPPC +

Mr. Jan M. Bult, President & CEO, PPTA, opened the well-attended IPPC 2018 by welcoming the more than 300 attendees to Budapest. PPTA thanks its sponsors who help make the IPPC possible every year. Download the IPPC app (available in the Apple AppStore and Android Market/Google Play) for Apple or Android devices) for a complete list of sessions and many of the presentations.

GLOBAL INITIATIVE DRAWS ATTENTION TO RARE DISEASE TREATMENTS +

Budapest, HUNGARY (March 13)– The Plasma Protein Therapeutics Association (PPTA) today unveiled its new global initiative, “How Is Your Day?” –Making the difference with plasma proteins, in conjunction with the annual International Plasma Protein Congress. “How Is Your Day?” will focus on differentiating these unique therapies from traditional pharmaceuticals and building awareness of the value they provide for people living with rare, life-threatening, chronic, and genetic diseases.